Avrobio posts positive data on gene therapy for rare diseases

Feb. 8, 2021

Avrobio, a clinical-stage gene therapy company focused on genetic disease, posted positive clinical data from its gene therapy trials in three different rare lysosomal diseases: Fabry, Gaucher type 1 and cystinosis.

The Cambridge, Mass.-based company now has 13 patients dosed across three clinical programs.

The data from the phase 2 FAB-GTstudy assessing AVR-RD-01, an investigational ex vivo lentiviral gene therapy for Fabry disease, showed complete clearance of toxic substrate Gb3 in the kidney biopsy of the first patient dosed with the plato gene therapy platform. The patient was dosed 3 1/2 years ago. Avrobio's proprietary platform includes a state-of-the-art lentiviral vector, a personalized conditioning regimen with precision dosing and an automated, closed manufacturing process intended to deliver potent and consistent drug product from manufacturing sites worldwide at commercial scale.

Avrobio also announced six-month data from the first patient dosed in the Phase 1/2 trial of AVR-RD-02, an investigational ex vivo lentiviral gene therapy for Gaucher disease type 1, showing plasma chitotriosidase and the toxic metabolite lyso-Gb1 – key biomarkers of Gaucher disease – had both dropped sharply below the patient’s baseline levels achieved on enzyme replacement therapy (ERT) before gene therapy was administered. Additionally, all three cystinosis patients in the investigator-sponsored Phase 1/2 trial of AVR-RD-04 show strong data across multiple measures and remain off cysteamine pills and eye drops, with trial enrollment completion expected this year.

Read the press release